Ontology highlight
ABSTRACT:
SUBMITTER: Nguyen L
PROVIDER: S-EPMC7391607 | biostudies-literature | 2020 Feb
REPOSITORIES: biostudies-literature
Neuron 20191209 4
The intronic C9orf72 G4C2 expansion, the most common genetic cause of ALS and FTD, produces sense- and antisense-expansion RNAs and six dipeptide repeat-associated, non-ATG (RAN) proteins, but their roles in disease are unclear. We generated high-affinity human antibodies targeting GA or GP RAN proteins. These antibodies cross the blood-brain barrier and co-localize with intracellular RAN aggregates in C9-ALS/FTD BAC mice. In cells, α-GA<sub>1</sub> interacts with TRIM21, and α-GA<sub>1</sub> tr ...[more]